A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China

Novartis Pharmaceuticals80 enrolled

Overview

The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients. Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

LNP023DRUG

Capsules for oral administration

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

Inclusion criteria For Cohort 1, Patient who meets all the following criteria can be included in this study: 1. Age ≥ 18 years at the time of signing the ICF; 2. Patient with a documented diagnosis of PNH; 3. Patient who has never received complement inhibitor therapy; 4. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF; 5. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible. 6. Patient who has signed the ICF. For Cohort 2, Patient who meets all the following criteria can be included in this study: 1. Age ≥ 18 years at the time of signing the ICF; 2. Patient with a documented diagnosis of PNH; Patients who have been receiving stable treatment with C5 complement inhibitors for at least three months prior to enrollment; 3. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF; 4. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; 5. If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible. 6. Patient who has signed the ICF. Exclusion criteria For Cohort 1 and Cohort 2, patients who meet any of the following criteria will meet the exclusion criteria for this study: 1. Participating in an interventional PNH clinical study; 2. Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose; 3. Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose; 4. Documented with a history of HIV infection; 5. Women who are pregnant or breastfeeding or intending to conceive during the study period; 6. Existence of bone marrow failure (reticulocytes \< 100 × 109/L, platelets \< 30 × 109/L, and neutrophils \< 0.5 × 109/L) determined by the investigator; 7. Other conditions that are not suitable for participating in the study, in the judgment of the investigator.

Locations (9)

Novartis Investigative Site

Guangzhou, Guangdong, China

RECRUITING

Novartis Investigative Site

Shijiazhuang, Hebei, China

RECRUITING

Novartis Investigative Site

Zhengzhou, Henan, China

Outcomes

Primary Outcomes

Change from baseline in hemoglobin (Hb) levels at designated time points

Assessment of the Hematological Response to iptacopan.

Time frame: Baseline, 12 months

Secondary Outcomes

Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: 12 months

Duration of Hb level ≥ 12 g/dL within a 12-month

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: 12 months

Change from baseline in LDH Levels after iptacopan initiation

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: Baseline, 12 Months

Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: 12 months

Number of participants having LDH normalization before and after iptacopan initiation

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: 12 monhts

Duration of LDH level ≤ 1.5 x ULN within 12 months

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: 12 months

Change from baseline in ARC Levels after iptacopan initiation

Central Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111 novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Novartis Investigative Site

Wuhan, Hubei, China

RECRUITING

Novartis Investigative Site

Nanchang, Jiangxi, China

RECRUITING

Novartis Investigative Site

Changchun, Jilin, China

RECRUITING

Novartis Investigative Site

Tianjin, China

RECRUITING

Novartis Investigative Site

Tianjin, China

RECRUITING

Novartis Investigative Site

Wuhan, China

RECRUITING

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: Baseline, 12 monhts

Number of participants having ARC normalization before and after iptacopan initiation

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: Baseline, 12 months

Change from baseline in bilirubin after iptacopan initiation

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: Baseline, 12 months

Number of participants with hepatosplenomegaly before and after iptacopan initiation

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: Baseline, 12 months

Number of participants with a positive coomb's test after iptacopan initiation

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: 12 months

Number of participants with PNH related signs or symptoms before and after iptacopan treatment

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: Baseline, 12 months

Change from baseline in PNH clone value

Change from baseline in flow cytometry results to assess the impact of iptacopan on Treatment-Related Outcomes.

Time frame: Baseline, 12 months

Rate, reasons (complement activation conditions (CAC), missed doses, etc.), duration, and interventions for breakthrough hemolysis (BTH)

Assessing the Impact of iptacopan on Treatment-Related Outcomes

Time frame: 12 months

Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale after iptacopan initiation

FACIT is a 40-item measure that assesses self-reported fatigue and its impact upon daily activities and function to assess the Impact of iptacopan on Treatment-Related Outcomes. The minimum value is 0 and maximum value is 52, and higher scores mean a worse outcome.

Time frame: Baseline, 12 months

Work Productivity and Activity Impairment Questionnaire-Specific Health Problems (WPAI-SHP) at each visit, change from baseline in WPAI-SHP after iptacopan initiation

WPAI-SHP is a tool used to measure the impact of health problems on work productivity and regular activities. The scale includes several components. Absenteeism: This measures the percentage of work time missed due to a specific health problem. Presenteeism: This measures the percentage of impairment while working due to the health problem. Work productivity loss: This combines absenteeism and presenteeism to measure overall work impairment. Activity impairment: This measures the percentage of impairment in regular activities due to the health problem. The scores are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity, which means worse outcomes.

Time frame: Baseline, 12 months

Adverse events (AEs) and Serious adverse events (SAEs) occurring during treatment with iptacopan

Assessing the safety of iptacopan

Time frame: 12 monhts

Treatment regimen for PNH 12 months before iptacopan treatment; reasons of discontinuation of previous complement inhibitor;

Assessing the impact of iptacopan on disease management

Time frame: Baseline

Use of concomitant medications (e.g., corticosteroids, androgen, immunosuppressants, anti-coagulants, etc.) for PNH and PNH-related complications (e.g. thrombosis, renal failure, etc.) before and after iptacopan treatment;

Assessing the impact of iptacopan on disease management

Time frame: Baseline, 12 months

Duration of iptacopan treatment, reasons for discontinuation, switching to other treatments, proportion of patients who switch from iptacopan to other treatments;

Assessing the impact of iptacopan on disease management

Time frame: 12 monhts

Number of participants with blood transfusion before and after iptacopan treatment, duration and number of units of blood transfusion due to BTH

Assessing the impact of iptacopan on disease management

Time frame: Baseline, 12 months

Number of participants hospitalized, outpatient, emergency room and intensive care unit (ICU) visits related to PNH

Assessing the impact of iptacopan on healthcare resource utilization

Time frame: 12 monhts

Length of inpatient stay related to PNH

Assessing the impact of iptacopan on healthcare resource utilization

Time frame: 12 months